Asia-wide Study for Acute Lymphoblastic Leukemia in Children with Down Syndrome

JRCT ID: jRCTs041180161

Registered date:29/03/2019

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Acute lymphoblastic leukemia in Down syndrome children
• Date of first enrollment: 21/08/2018
• Target sample size: 60
• Countries of recruitment: Singapore,Japan,China,Japan,Taiwan,Japan,Thailand,Japan
• Study type: Interventional

Intervention(s)

Induction therapy, 5 weeks
Consolidation therapy, 8 weeks
Re-induction therapy, 3 weeks
Interim maintenance therapy, 4 weeks
Maintenance therapy 48+72 weeks

Outcome(s)

• Primary Outcome: Event Free Survival

Secondary Outcome

- Overall survival, Disease free survival, Induction failure, Complete remission rate after induction (IA) of each risk group and whole group, Cumulative incidence of relapse, Continuous CR (CCR)
- Treatment related mortality at end of induction and overall,
Morbidity, Mortality, Incidence of infectious and metabolic complications Incidence of adverse events Incidence of secondary neoplasms
- MRD at day 15

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: <= 20age old
• Gender: Both

Include criteria

Participants are eligible to be included in the study only if all of the following criteria apply:
1. Down syndrome diagnosed clinically or cytogenetically (including Mosaic Down)
2. Newly diagnosed ALL according to WHO 2016 classification.
3. Age < 21 years old at time of enrollment.
4. ECOG performance status (PS) score of 0-2.
5. Written informed consent obtained from legally acceptable representatives.

Exclude criteria

Participants are excluded from the study if they have any of the following criteria:
1. Second malignancy.
2. Philadelphia positive ALL.
3. Mature B-ALL.
4. Mixed phenotype acute leukemia.
5. Any previous treatment with cytotoxic chemotherapy excluding treatment for TAM or radiation therapy. Patient pre-treated with short term steroid (< 7 days of duration within last 1 month prior to treatment start) can be enrolled into this study.
6. Renal dysfunction with creatinine >2x upper limit of normal (ULN). Patients whose creatinine has improved to <2x ULN before treatment commencement can enrol subject to discretion of site PI.
7. Liver dysfunction with direct bilirubin > 5x ULN.
8. Any serious uncontrolled medical condition or impending end organ dysfunction that would impair the ability of the subject to receive protocol therapy, including:
a. History of coronary arterial disease, cardiomyopathy, heart failure, arrhythmia (other than sinus arrhythmia) or severe cardiac malformation which with residual abnormalities or requires further major corrective surgery within 2 years.
b. Ongoing uncontrolled hypertension.
c. Ongoing uncontrolled diabetes mellitus.
d. Ongoing uncontrolled infection.
e. History of congenital or acquired immunodeficiency including HIV infection.
f. History of interstitial pneumonia, pulmonary fibrosis, bronchiectasis or severe pulmonary emphysema.
g. CNS hemorrhage.
h. Psychiatric disorder.
i. Other concurrent active neoplasms.
9. Pregnant or lactating women.
10. Doubtful compliance or ability to complete study therapy due to financial, social, familial or geographic reason, or in the judgement of site investigator.

Related Information

• Primary Sponsor: Okamoto Yasuhiro
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development
• Secondary ID(s): NCT03286634